Symposium Q&A from Daniel Leffler, MD, MS

Q. How do you differentiate between a “Super sensitive” celiac and refractory celiac? Are there treatment options for either?

A. Many people believe that type 1 refractory celiac disease occurs when people are so highly sensitive that there is no feasible way to be sufficiently gluten-free. There is no clinical way to differentiate these conditions, if they are indeed separate, so at this time both are treated mostly with budesonide (a steroid with low systemic absorption).

Q. When diagnosed I was told not to drink “gluten removed” beer. With KumaMax, would that change?

Is KumaMax only for very small cross contamination? Can it break down anything other than gluten?

A. We don’t really know how potent KumaMax will be at degrading gluten, but given the published literature, it seems to be very effective and may allow for a gram or more of gluten to be eaten safely, such as a bottle of regular beer. This would allow for consumption of most low-gluten foods without toxicity. As far as we know, KumaMax only breaks down gluten, not other food proteins.

Q. Do you foresee a way for successful new therapies to prevent symptoms of gluten ingestion during a gluten challenge (so those challenged are less sick)?

A. Yes, I think any successful therapy for celiac disease should be able to prevent symptoms during gluten challenge. This was shown with both larazotide and ALV003 in separate gluten challenge studies. Larazotide was able to prevent symptoms and elevation in tTG with gluten challenge, and ALV003 was able to prevent damage to the small intestine.

Q. What are the risks involved in being part of a research study for celiac patients who will need to ingest gluten? Will it increase chances for comorbidities?

A. I think is worth remembering that before we had good blood tests for celiac disease, gluten challenge was part of the diagnosis of ALL patients with celiac disease, including children, and that these gluten challenges often went on for months. These generations of patients all did fine, so this gives us great confidence that the 2-6 week gluten challenges used in research are indeed safe.

Q. What is the timeline for Larazotide or any of the other intraluminal (in the cavity of the small intestine) therapies?

A. This is always difficult to predict, but if larazotide is successful in its upcoming study, it could be available in 2-3 years. Everything else is likely to be 5 years later than that.

Q. Does the Nexvax vaccine also cause intestinal damage as it induces symptoms?

A. This is currently unknown but will be looked at in their upcoming phase 2 study.